The NeurologyLive® neuromuscular disease clinical focus page offers updates and information related to the clinical care and management of individuals with neuromuscular diseases such as amyotrophic lateral sclerosis, Duchenne muscular dystrophy, cerebral palsy, and spinal muscular atrophy, as well as rare diseases. This page consists of news, podcasts, and videos about the latest FDA actions, clinical guideline updates, interviews with physicians, study and clinical trial findings, and more.
July 30th 2025
Capricor Therapeutics navigates FDA challenges for deramiocel, aiming to advance treatment for DMD-related cardiomyopathy with promising trial data.
Enhancing Multidisciplinary Care for Neuromuscular Disorders: Amanda Peltier, MD
March 25th 2024The professor of neurology at Vanderbilt University Medical Center talked about the importance of multidisciplinary care centers to improve accessibility and quality of care for patients with neuromuscular disorders. [WATCH TIME: 5 minutes]
Overviewing Phase 2 SYNAPSE-SMA Trial of NMD670 for Spinal Muscular Atrophy: Jorge Quiroz, MD, MBA
March 22nd 2024The chief medical officer of NMD Pharma provided perspective on a newly launched phase 2 study assessing NMD670, a first-in-class neuromuscular transmission enhancer, in patients with spinal muscular atrophy type 3. [WATCH TIME: 5 minutes]
Flaws in Assessment Scales for Neuromuscular Conditions: Laurent Servais, MD, PhD
March 20th 2024The professor of pediatric neuromuscular diseases at the University of Oxford provided commentary on the commonly used scales to assess neuromuscular function and where inconsistencies have been observed. [WATCH TIME: 7 minutes]
The Glymphatic Pathway: Evaluating Neurological Conditions From a New Perspective
March 20th 2024This complex waste clearance pathway within the CNS plays a crucial role in maintaining brain homeostasis, as a mechanism of protein waste removal alongside autophagy and protein aggregate ubiquitination.
Expanding the Access and Possibilities of Care for Adults With Neuromuscular Disorders
March 11th 2024Amanda Peltier, MD, a professor of neurology at Vanderbilt University Medical Center, spoke on current care needs for adults with neuromuscular disorders and the realistic ways to enhance clinical care going forward.
Adding to the Literature of KIF5A in ALS Pathogensis: Devesh Pant, PhD
March 11th 2024The instructor in the department of cell biology at Emory University provided context on a presentation at MDA 2024 regarding mutations in Kif5a in familial forms of amyotrophic lateral sclerosis. [WATCH TIME: 4 minutes]